Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients-Reference-Cited by-同舟云学术

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Published:2016-11-04 Issue:1 Volume:6 Page:
ISSN:2045-2322
Container-title:Scientific Reports
language:en
Short-container-title:Sci Rep

Author:

Masat Elisa, ,Laforêt Pascal,De Antonio Marie,Corre Guillaume,Perniconi Barbara,Taouagh Nadjib,Mariampillai Kuberaka,Amelin Damien,Mauhin Wladimir,Hogrel Jean-Yves,Caillaud Catherine,Ronzitti Giuseppe,Puzzo Francesco,Kuranda Klaudia,Colella Pasqualina,Mallone Roberto,Benveniste Olivier,Mingozzi Federico,Bassez G.,Bedat-Millet A. L.,Behin A.,Eymard B.,Leonard-Louis S.,Stojkovic T.,Canal A.,Decostre V.,Bouhour F.,Boyer F.,Castaing Y.,Chapon F.,Cintas P.,Durieu I.,Echaniz-Laguna A.,Feasson L.,Furby A.,Hamroun D.,Ferrer X.,Solé G.,Froissart R.,Piraud M.,Germain D.,Benistan K.,Guffon-Fouilhoux N.,Journel H.,Labauge P.,Lacour A.,Levy A.,Magot A.,Péréon Y.,Minot-Myhié M. -C.,Nadaj-Pakleza A.,Nathier C.,Orlikowski D.,Pellegrini N.,Petiot P.,Praline J.,Lofaso F.,Prigent H.,Dutry A.,Renard D.,Sacconi S.,Desnuelle C.,Salort-Campana E.,Pouget J.,Tiffreau V.,Vincent D.,Zagnoli F.

Publisher

Springer Science and Business Media LLC

Subject

Multidisciplinary

Link

http://www.nature.com/articles/srep36182.pdf

Reference65 articles.

1. van der Ploeg, A. T. & Reuser, A. J. Pompe’s disease. Lancet 372, 1342–1353, doi: 10.1016/S0140-6736(08)61555-X (2008).

2. Hirschhorn, R. a. R. AJJ. In The Metabolic and Molecular Basis of Inherited Disease (ed A. L. Beaudet, C. R. Scriver, W. S. Sly, D. Valle ) 3389–3420 (McGraw-Hill, 2001).

3. Kroos, M., Hoogeveen-Westerveld, M., van der Ploeg, A. & Reuser, A. J. The genotype-phenotype correlation in Pompe disease. Am J Med Genet C Semin Med Genet 160C, 59–68, doi: 10.1002/ajmg.c.31318 (2012).

4. Kishnani, P. S. et al. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab 99, 26–33, doi: 10.1016/j.ymgme.2009.08.003 (2010).

5. Orlikowski, D. et al. Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease. Neuromuscul Disord 21, 477–482, doi: 10.1016/j.nmd.2011.04.001 (2011).

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